Efficacy and Safety of Pasireotide Treatment in Acromegaly: A Systematic Review and Single Arm Meta-Analysis

Abstract Background Acromegaly is an uncommon chronic endocrine disorder which is associated with considerable comorbidities. Many patients fail to achieve biochemical control with current medical therapies, including surgery and first-generation somatostatin analogs. We aimed to perform a systematic review and single-arm meta-analysis to evaluate the efficacy of the multi-receptor ligand somatostatin analog Pasireotide in patients with active or uncontrolled acromegaly. Methods We systematically searched PubMed, Embase, and Cochrane databases for studies that assessed the efficacy of Pasireotide in patients with acromegaly and reported the outcomes of (1) biochemical control and its composite indicators; (2) normalized IGF1 level and (3) low GH levels. For the statistical analysis, we used R software. Results We included nine studies with a total of 590 patients: four clinical trials and five observational cohorts. The overall population consisted of inadequately controlled or active acromegaly patients. After a follow-up of 12 months, the overall population achieved a biochemical control rate of 26.50% (95% CI: 14.87-42.66). The prevalence of normalized IGF1 and low GH levels was 36.27% (95% CI 29.15-43.39) and 34.76% (95% CI 24.58-44.95), respectively. Additionally, biochemical response rates were sustained throughout the extension phase of these studies. In a pooled analysis including four studies with extension phase results, the prevalence of biochemical control rate in the overall population was 29.03% (95% CI: 11.49–46.58) with 76 events out of 281 patients. The most commonly reported adverse events were gastrointestinal disturbances in 31.26% (95% CI: 7.44–72.01) and hyperglycemia in 29.55% (95% CI: 21.80–37.29) of patients. The incidence of diabetes mellitus significantly increased after pasireotide treatment, with a rate of 23.36% (95% CI: 19.58-27.13). Conclusion Pasireotide demonstrates biochemical control in patients with active or uncontrolled acromegaly. Although a high rate of hyperglycemic adverse events and diabetes mellitus related to the treatment were observed, most of them were manageable.